OUTLOOK NEGLECTED DISEASES

NATURE|Vol 449|13 September 2007

Patent sense
Protecting intellectual property saves lives in the developing world, argues Paul Herrling.
any diseases are endemic in the developing world, yet for a number of these there are few safe and effective treatments. This lack of medicines results from an industrial model that has been in place for more than 50 years. Basic scientific research carried out in the public sector is translated into life-saving medicines mainly by pharmaceutical companies. This is a lengthy, onerous and expensive process — taking about 15 years and costing Non-profit institutes funded by industry carry out R&D on drugs for neglected diseases. hundreds of millions of dollars per drug — and comes with a high risk of failure. Nevertheless, are based, to a large extent, on innovation. cannot use a patent-protected invention in more than 90% of new molecular entities dis- A patent is defined as a grant by the state of their studies — that the system is a considercovered and developed as medicines between exclusive rights for a limited time in respect able barrier to further innovation. To prevent 1990 and 1999 originated from pharmaceutical of a new and useful invention. These rights such abuse of patents, several countries have companies1,2. usually imply that, for a limited time, only the implemented the ‘research exemption’, which Drug firms may be the main source of new innovator, or a person or entity licensed by allows scientists to use patent-protected techtherapies, but they remain commercial enti- the innovator, can sell products based on the nology freely for their research provided they ties that can invest the considerable resources invention. This offers the innovator an oppor- do not exploit it commercially. In light of these required to translate basic science into an tunity to recover the investment needed to issues, the protection of intellectual property effective medication only when there is a rea- develop the invention into a practical product. with patents is crucial for pharmaceutical comsonable chance of financial return. Without this incentive, important discoveries panies to discover and develop new drugs for There is little opportunity to get an adequate would never be developed into useful prod- the developing world. return on investment for infectious diseases ucts. Modern patent law provides protection such as tuberculosis (TB), for 20–25 years, which Neglected no longer dengue fever, malaria, “In the absence of a patent, should be compared with In the past decade or so, the drug industry has leishmaniasis and African the 15 years, on average, formed partnerships with the public sector, the only way inventors can needed for the discov- generating pipelines of early-stage potential trypanosomiasis (sleeping protect their inventions is ery and development of medicines for certain neglected diseases. These sickness), which mainly a new drug. In return for partnerships include the Global Alliance for TB affect people living in through total secrecy.” these rights, the innova- Drug Development, the Drugs for Neglected resource-poor regions. In other words, market mechanisms fail in tor discloses a description of the invention Diseases Initiative (DNDi) and the Medicines these cases, and there is insufficient drug- that allows other experts to reproduce the for Malaria Venture. In 2004, 63 new drugs discovery research and development (R&D) key findings. This process is firmly based on were being pursued by this approach3. for these common infectious diseases. the premise that knowledge is gained only Most of these drugs originate from R&D through a full understanding and apprecia- programmes in pharmaceutical companies No secrets tion of previously published advances. and are patented accordingly. But these patents Some organizations interested in improving In the absence of a patent, the only way inven- are not used to enforce unaffordable prices in access to medicines in the developing world, tors can protect their inventions is throughtotal the developing world or to prevent manufacsuch as Médecins Sans Frontières and Oxfam, secrecy, which is counter to furthering innova- turers from selling generic versions of these think that a major impediment to affordable tion, a fact often ignored by those who con- drugs in developing regions. medicines is the patent system. But this is sider that patents prevent research. It is only An example of this is Coartem, an not the case. This system protects intellec- when patents are used excessively to protect artemisinin-based combination therapy. One tual property in countries whose economies information — to the extent that researchers of the most effective treatments for malaria

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NATURE|Vol 449|13 September 2007

NEGLECTED DISEASES OUTLOOK

tries at cost price. A generic version of the drug is being manufactured by Indian pharmaceutical companies and sold in the developing world. Another example is a new artemisininbased combination therapy for malaria, developed by sanofi-aventis in collaboration with the DNDi. This treatment, also on the World Health Organization’s list of essential medicines5, will be available in most of sub-Saharan Africa next year. Sanofi-aventis has set aside its patent rights and will supply the medicine at cost price to the poorest populations in countries in which malaria is endemic. In general, drugs developed by pharmaceutical companies as part of public–private partnerships are patented by industry, as it is the discoverer. But they are exclusively licensed for treating neglected diseases in agreements stipulating that the drugs will be available at cost price to the developing world.

Necessary system
So why are patents even necessary? There are three main reasons. First, commercial entities, including drug companies, can allocate resources to non-profit projects only if they are financially sound. Research- and innovationbased companies earn sufficient returns on their R&D investments only if they are permitted a marketing-exclusivity period — accorded by patents — for their innovative products. In countries without this protection in place, the drug industry is not research intensive and innovation based. Indeed, this was the case in India while there was no patent protection for new pharmacologically active molecules. The randomness of evolution provides a sec-

at present4, Coartem is included on the list of essential medicines by the World Health Organization5. One of its components was discovered by Chinese scientists, and this was then clinically characterized, and developed and produced as a combination therapy, by Novartis. Coartem is patented in 49 countries but is available at cost price to patients in all countries in which malaria is endemic. In 2006, Novartis delivered more than 62 million treatments of Coartem to more than 30 coun-

ond reason for patenting drugs for neglected diseases. Nature does not distinguish between the biology of diseases of the poor and the rich. So therapeutic molecules or pathways that are targeted by drugs for neglected diseases might also be relevant for treating diseases that affect people in more affluent regions. For example, the Novartis Institute for Tropical Diseases in Singapore takes all molecules that have activity against dengue virus and systematically tests them against the West Nile and hepatitis C viruses, which belong to the same family as dengue and cause disease also in developed countries. If a molecule has activity against dengue virus or another family member, it is developed and patented. Considerable financial returns could be generated in the developed world, and a portion of these is earmarked for refinancing, and for providing long-term sustainability to, the institute’s nonprofit initiatives. A third reason to patent such drugs relates to emerging economies such as those of Brazil (see page 180) and India. In these countries, there are populations of very poor patients, and the non-profit model would certainly apply. But in the same countries, there are growing numbers of more affluent patients, who are increasingly able to buy their medication, either directly or through health insurance. For these patients, the company that developed the medicine could expect to generate revenue as a result of patent protection. Such differential pricing within a country (see page 176) would encourage further innovation not only by global companies but also by local enterprises. This model would require legislation that prohibits copying before patents have expired but that allows generic-drug production after patent expiry. For the foreseeable future, the discovery and development of new medicines will be driven almost exclusively by commercial pharmaceutical companies. The only way that these firms can remain viable is through a robust intellectual-property protection system. This system, therefore, contributes to saving lives. Without it, there would be few new drugs for any disease, regardless of whether it afflicts the rich or the poor. ■
Paul Herrling is head of Corporate Research, Novartis International, Basel, Switzerland.
1. Reichert, J. M. & Milne, C. P. Am. J. Ther. 9, 543–555 (2002). 2. DiMasi, J. A., Hansen, R. W. & Grabowski, H. G. J. Health Econ. 22, 151–185 (2003). 3. Moran, M. PLoS Med. 2, e302 (2005). 4. Mutabingwa, T. K. et al. Lancet 365, 1474–1480 (2005). 5. World Health Organization Model List of Essential Medicines. www.who.int/medicines/publications/EML15. pdf (2007).

K. PRINSLOO/AP

NOVARTIS

Aid organizations such as Médecins Sans Frontières say that patents block access to medicines.
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